Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
This research study is testing a new medicine called zeleciment basivarsen (DYNE-101) for people with Myotonic Dystrophy Type 1 (also known as DM1 or Steinert Disease). The main goal is to find out if this medicine is effective at treating the condition, how safe it is, and if people can take it without too many side effects. Participants will be split into groups; one group will receive the new study medicine and the other will receive a dummy medicine (called a placebo) that looks the same but contains no active drug. This comparison helps us understand the true effects of the new medicine. The study will last for a total of nearly a year and a half, including initial checks, the treatment period, and follow-up.
At a glance
What is this study about?
This study is investigating a new potential treatment for Myotonic Dystrophy Type 1 (DM1), a condition that can affect muscles and other parts of the body. The experimental medicine is named zeleciment basivarsen, also known by its research code DYNE-101. Researchers want to understand if this medicine can make a positive difference for people living with DM1. They will specifically look at three main things: how well it works (its 'efficacy'), how safe it is for people to take, and whether people can tolerate it without experiencing difficult side effects.
To properly test the medicine, the study will involve a 'placebo-controlled' period. This means some participants will receive the actual study medicine, and others will receive a 'placebo' – a substance that looks exactly like the medicine but has no active ingredients. Comparing results between these two groups helps doctors determine if any changes or improvements are genuinely due to the new medicine or if they might have happened anyway. After this main treatment phase, there's also a period for long-term follow-up.
An important safeguard in this study is an Independent Data Monitoring Committee. This group is made up of experts who are not involved in running the study. They will regularly check the safety information gathered from all participants. Their role is to ensure everyone's well-being throughout the trial and to make recommendations if any safety concerns arise. This independent check adds an extra layer of protection for participants.
Key takeaways
- This study evaluates a new medicine (DYNE-101) for Myotonic Dystrophy Type 1.
- It aims to check the medicine's effectiveness, safety, and how well people tolerate it.
- Participation involves screening, a treatment period (with either the drug or a placebo), and follow-up.
- An independent committee is overseeing participant safety throughout the study.
- The total time commitment for the study is approximately 1.5 years.
Who may be eligible?
To join this study, your doctor will need to confirm you have Myotonic Dystrophy Type 1 (DM1) with a specific genetic test result showing a 'trinucleotide repeat size' greater than 100. This genetic test confirms the type of DM1. You must also be able to walk 10 meters and get up from a chair 5 times by yourself. You can use walking aids like insoles or ankle supports if they don’t go above your ankle.
Your body mass index (BMI) needs to be under 35. You cannot join if you have been diagnosed with the serious 'congenital' form of DM1 or if you've had major surgery in the last 12 weeks, unless it was for a pacemaker or defibrillator. Also, if you've recently taken certain diabetes or weight-loss medicines (like semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide), you might need to wait a specific period before being able to participate.
There might be other health requirements or reasons why you can or cannot join. The study team will review all your medical information carefully to see if this study is suitable for you.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Do I have a confirmed diagnosis of Myotonic Dystrophy Type 1 (DM1) from a genetic test?
- Can I walk 10 meters and stand up from a chair 5 times on my own (with minor aids if needed)?
- Is my Body Mass Index (BMI) below 35?
- Have I avoided certain GLP-1/incretin medications (like Ozempic, Trulicity) recently?
- Have I had any major surgery in the last 12 weeks (excluding pacemaker/defibrillator)?
- Have I been diagnosed with 'congenital' DM1 (this would exclude me)?
- Am I aged 16 years or older?
What does participation involve?
If you decide to take part, the study will have different stages. First, there's a 'Screening' period which can last up to 8 weeks. During this time, you'll have various tests and checks to make sure you meet all the criteria for joining the study. After screening, there’s a main 'Placebo-Controlled Period' which lasts for 48 weeks (just under a year). During this time, you will receive either the study medicine or the placebo. Finally, there's a 'Long-Term Extension Period' of 24 weeks (about 5 and a half months) to continue tracking your health and the medicine's effects. You will have regular visits to the clinic for assessments, which will include examinations, blood tests, and other specific checks related to your DM1. The exact number of visits will be explained by the study team, but throughout the entire study, you can expect to be involved for about a year and a half.
Potential risks and benefits
Locations (15)
- UCSD - Altman Clinical and Translational Research InstituteVerified postcodeLa Jolla, United States· Recruiting
- Rare Disease Research, LLCVerified postcodeAtlanta, United States· Recruiting
- IU Health Neuroscience CenterVerified postcodeIndianapolis, United States· Recruiting
- Roy Blunt NextGen Precision Health InstituteVerified postcodeColumbia, United States· Recruiting
- Rare Disease Research, LLCVerified postcodeHillsborough, United States· Recruiting
- The University of Texas Health Science Center at San AntonioVerified postcodeSan Antonio, United States· Recruiting
- UZ LeuvenVerified postcodeLeuven, Belgium· Recruiting
- Rigshospitalet, (Neuromuscular Clinic and Research Unit, Department 8077)Verified postcodeCopenhagen, Denmark· Recruiting
- Pitié-Salpêtrière HospitalVerified postcodeParis, France· Recruiting
- LMU Klinikum der Universität München Neurologische Klinik und Poliklinik Friedrich-Baur-Institut Campus InnenstadtVerified postcodeMunich, Germany· Recruiting
- Charité Universitätsmedizin Berlin Campus-Buch Muscle Research UnitVerified postcodeBerlin, Germany· Recruiting
- National Hospital Organization Osaka Toneyama Medical CenterUnverifiedToyonaka-Shi, Japan· Recruiting
Common questions
What is Myotonic Dystrophy Type 1 (DM1)?
DM1 is a genetic condition that mainly causes muscle problems, like weakness and stiffness, but can also affect other parts of the body.
What is zeleciment basivarsen (DYNE-101)?
It's a new medicine being tested in this study to see if it can treat Myotonic Dystrophy Type 1.
What does 'placebo-controlled' mean?
It means some people will get the real medicine, and others will get a 'dummy' medicine without active ingredients. This helps researchers compare the effects.
How long will I be in the study?
The study involves a screening period, a treatment period, and a follow-up period, lasting about one and a half years in total.
Will I know if I'm getting the real medicine or the placebo?
During the main part of the study for ethical reasons, neither you nor your study doctor will know who is getting which. This is called 'blinding'.
How to find out more
Dyne Clinical Trials
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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